Assessment of Outcomes Associated With the Use of Newly Approved Oncology Drugs in Medicare Beneficiaries

Abstract

Key Points Question What are the real-world outcomes for Medicare patients with metastatic cancer receiving recently approved oncology drugs and how do they compare with pivotal clinical trial outcomes? Findings In this retrospective cohort study, outcomes were compared between clinical trial participants and treated Medicare patients across 22 cancer drugs approved by the US Food and Drug Administration for 29 indications. Median duration of therapy and overall survival among treated Medicare patients were generally shorter than among trial participants, and dose reductions were common among Medicare patients. Meaning Pivotal clinical trials may provide inadequate data for the purpose of clinical decision-making among Medicare beneficiaries with advanced cancer.

Association Between First-Line Immune Checkpoint Inhibition and Survival for Medicare-Insured Patients With Advanced Non–Small Cell Lung Cancer

“…In this population, patients who received immunotherapy or chemoimmunotherapy were older, were more likely to be female, and had higher baseline mortality risk than those given chemotherapy alone. Survival after first-line immunotherapy was much shorter than reported in registrational clinical trials; this finding is consistent with a recently published analysis of patients diagnosed with multiple types of cancer from 2008 to 2013, 45 though the latter analysis cohort was too remote to assess the effectiveness of immunotherapy for lung cancer. In both multivariable- and propensity score–adjusted models that controlled for baseline differences in patients’ clinical and sociodemographic characteristics, overall survival in this cohort was similar among patients who received single-agent immunotherapy, combination chemoimmunotherapy, or chemotherapy only.…”

“… 50 Although a difference between survival in real-world populations and clinical trial participants could be due to competing causes of mortality in some contexts, most deaths in our cohort would be attributable to lung cancer, as observed in a recent SEER-Medicare analysis of patients with more remote diagnoses. 45 In addition, this study focused specifically on the association between first-line therapy and outcomes, but second-line immunotherapy may well have provided benefit to some patients in the cohort who received first-line chemotherapy, diminishing the association of first-line therapy with overall survival.…”

Nutritional support during the hospital stay reduces mortality in patients with different types of cancers: secondary analysis of a prospective randomized trial

“…Nevertheless, our results support the clinical relevance of simultaneously addressing patients' oncological and nutritional needs, and provide a possible explanation to the recently reported discrepancies in outcomes for patients enrolled in clinical trials and those in registries. [29] Considering that patients with cancer with comorbidities, including malnutrition, are less likely to be offered to participate to a clinical trial, [30] prevention and treatment of malnutrition may confer additional benefits. Also, J o u r n a l P r e -p r o o f concurrent care may enhance patients with cancer' quality of life, an issue frequently overlooked even under the protected umbrella of a clinical trial.…”

A Reliability Generalization Meta-Analysis of 17 Patient-Reported Outcome Measures for Positive Psychosocial Constructs in Children, Adolescents, and Young Adults with Cancer

“…It is now understood that patients in cancer trials are younger and healthier than patients in the real world. 32 In a similar manner, reliability of PROM scores may change in novel samples of patients, especially if there are differences in terms of age, race, gender, and other cultural factors. Reports of reliability in each new sample are therefore vital to understanding the variation in psychometric properties of a PROM.…”